Retinitis pigmentosa leads to a slow and irreversible loss of vision. X-Linked Retinitis Pigmentosa or simply the XLRP is a rare orphan disease resulting from a congenital genetic defect and one of the most common causes of blindness in young people with no treatment options currently available. Mutations in a gene located in the X chromosome, causes choroideremia, a rare disease in which the retina degenerates slowly over the years, eventually leaving patients blind.
Nightstarx Ltd (Nightstar), a biopharmaceutical spinout company of Oxford is developing gene therapies for inherited retinal diseases that cause blindness. The company had commenced enrolling and dosing subjects in a Phase I/II clinical trial to treat patients with X-Linked Retinitis Pigmentosa (XLRP) earlier this year and has now announced the money through a Series C round with $45M (€39.5M) which will be used to fund the upcoming Phase III trial with its lead candidate NSR-REP1 in choroideremia.
The biotech’s technology is based on the adeno-associated virus (AAV) vectors that deliver functional genes- speicifically- the codon-optimised copy of the retinitis pigmentosa GTPase regulator (RPGR) gene into cells of the eye of patients with mutations that affect their sight.
A previous Phase I/II trial in six patients where
only one eye was treated showed significant improvement in their vision three and a half years after receiving the therapy.The fundraising was backed by existing investors Syncona, the VC arm of the UK’s Wellcome Trust, and New Enterprise Associates (NEA), which were joined by two new names: Wellington Management and Redmile.
